The Benefit of 5% IVIG for Patients With Primary Immunodeficiency Disorders Who Experience Adverse Events on 10% IVIG Preparations

An Investigator Driven Observational Study to Determine the Benefit of Octagam 5% for Treatment of Patients Diagnosed With Primary Immunodeficiency Disorders (PID) on Intravenous Immunoglobulin (IVIG) Therapy That Experience Adverse Events (AEs) on Any 10% IVIG Preparation

Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration IVIG product.

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiency

Detailed Description

Patients with PID require life long immunoglobulin (Ig) replacement therapy with IVIG being the most common form. As more 10% IVIG products are FDA approved, the older and well characterized 5% IVIG products are becoming less used. Currently, the standard of care for patients who experience AEs on IVIG is to move to a subcutaneous (SCIG) delivery and product. This study will evaluate the AEs on a 10% product and octagam 5%. The study will enroll 15 patients after an AE on any 10% product who will then be infused with octagam 5% for six infusions. AEs will be documented and compared to the 10% product along with changes in biomarkers. The study data may document another therapeutic option for patients who experience AEs - SCIG and octagam 5%.

• Study Type: Observational
• Enrollment (Actual): 15

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Diagnosis of PID - specifically common variable immunodeficiency (CVID)

Description

Inclusion Criteria:

• Participants, or legal guardians with assent by underage children, will sign informed consent/assent and are willing to comply with all aspects of the study
• Diagnosis of CVID according IUIS Expert Committee
• Participants on a 10% product who experience AEs
• Ages between 10 and 75 years of age
• Participants on 10% IVIG therapy every 21±3 days or 28±3 days between 300 - 800 mg/Kg body weight

Exclusion Criteria:

• Acute infection requiring antibiotic therapy within 7 days prior to visit 1
• Presence of any condition that is likely to interfere with the evaluation of the study medication or satisfactory conduct of the trial
• History of anaphylactic or severe systemic reactions to human immunoglobulin
• IgA deficient patients with antibodies against IgA and a history of hypersensitivity
• Females who are pregnant or lactating

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5%	AEs will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The change in levels of inflammatory biomarkers associated with AEs between any 10% IVIG and octagam 5%		Levels will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
Safety Evaluations (complete blood count [CBC])	CBC	Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (Complete Metabolic profile[CMP])	CMP	Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (IgG trough level)	IgG trough level	Screening and prior to last infusion up to 24 weeks

Collaborators and Investigators

• Sponsor: IMMUNOe Research Centers

Publications and helpful links

General Publications

• Kaveri SV, Maddur MS, Hegde P, Lacroix-Desmazes S, Bayry J. Intravenous immunoglobulins in immunodeficiencies: more than mere replacement therapy. Clin Exp Immunol. 2011 Jun;164 Suppl 2(Suppl 2):2-5. doi: 10.1111/j.1365-2249.2011.04387.x.
• Geha RS, Notarangelo LD, Casanova JL, Chapel H, Conley ME, Fischer A, Hammarstrom L, Nonoyama S, Ochs HD, Puck JM, Roifman C, Seger R, Wedgwood J; International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. Primary immunodeficiency diseases: an update from the International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. J Allergy Clin Immunol. 2007 Oct;120(4):776-94. doi: 10.1016/j.jaci.2007.08.053.
• Deane S, Selmi C, Naguwa SM, Teuber SS, Gershwin ME. Common variable immunodeficiency: etiological and treatment issues. Int Arch Allergy Immunol. 2009;150(4):311-24. doi: 10.1159/000226232. Epub 2009 Jul 1. Erratum In: Int Arch Allergy Immunol. 2010;151(4):284. Dosage error in article text.
• Maarschalk-Ellerbroek LJ, Hoepelman IM, Ellerbroek PM. Immunoglobulin treatment in primary antibody deficiency. Int J Antimicrob Agents. 2011 May;37(5):396-404. doi: 10.1016/j.ijantimicag.2010.11.027. Epub 2011 Jan 26.

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2015
• Primary Completion (Actual): November 1, 2016
• Study Completion (Actual): September 1, 2017

Study Registration Dates

• First Submitted: March 31, 2015
• First Submitted That Met QC Criteria: November 7, 2017
• First Posted (Actual): November 13, 2017

Study Record Updates

• Last Update Posted (Actual): November 13, 2017
• Last Update Submitted That Met QC Criteria: November 7, 2017
• Last Verified: November 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases
• Other Study ID Numbers: IIS201401-PID